Octreotide, which is a synthetic analogue of somatostatin, inhibits endocrine and exocrine secretions.
Octreotide is approved by Health Canada for use in acromegaly, neuroendocrine tumours, pancreatic surgery, and emergency bleeding of gastroesophageal varices. Octreotide is also being used for other unapproved indications.
Given the growing use of octreotide and the availability of a long-acting somatostatin analogue (lanreotide), a review of the clinical and cost-effectiveness evidence on the approved uses and on six selected unapproved uses of octreotide is timely.
Methods and Results
Relevant meta-analyses or systematic reviews, randomized controlled trials (RCTs), controlled clinical trials, and economic analyses were identified. Eight-two RCTs were included. Meta-analysis was possible for outcomes in five indications. Data from the non-randomized controlled clinical trials were used for the assessment of harms only. For the economic analysis, a narrative synthesis of eight identified economic evaluations (four in a Canadian context) was conducted, and a budget impact analysis for five publicly funded drug plans was completed.
Implications for Decision Making
- Substantial uncertainty remains. An effect on mortality has not been observed. Octreotide improved surrogate markers of efficacy or short-term symptom control in patients with acromegaly, neuroendocrine tumours, esophageal bleeding, and bowel obstruction. For patients with hepatocellular carcinoma, pancreatic cancer, or refractory diarrhea related to HIV-AIDS or chemotherapy, no consistent clinical effect was found. Octreotide is not associated with substantial harm in the short term. The impact on health-related quality of life, the efficacy of short-acting versus long-acting octreotide, or the optimal duration of octreotide therapy is largely unknown.
- The use of octreotide in pancreatic surgery warrants consideration. Octreotide reduced the risk of some complications after pancreatic surgery and is more effective and less costly compared to placebo.
- A potential for increased expenditures exists. The coverage of unapproved indications could double the expenditures on octreotide for publicly funded drug plans. If only palliative care programs are funded, reimbursement for approved and unapproved indications results in a smaller budget increase.
This summary is based on a comprehensive health technology assessment available from CADTH’s web site (www.cadth.ca): Murphy G, Perras C, Desjardins B, Chen S, Moulton K, Jonker D, Perlman K, Pasieka J, Ezzat S, Cripps C, Mensinkai S, Skidmore B. Octreotide for endocrine, oncologic, and gastrointestinal disorders: systematic review and budget impact analysis.